1. A new modeling approach allowing prediction and comparison of the long-term outcomes of treatments for hemophilia B.
- Author
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Keep MV, Hoxer CS, Hemstock M, Groth AV, and Knight C
- Subjects
- Hemophilia B complications, Hemorrhage etiology, Hemorrhage prevention & control, Humans, Markov Chains, Recombinant Proteins therapeutic use, Time, Treatment Outcome, Factor IX therapeutic use, Factor X therapeutic use, Hemophilia B drug therapy, Polyethylene Glycols therapeutic use
- Abstract
Aim: To develop a modeling approach to compare clinical outcomes of nonacog beta pegol to a standard-acting factor IX (FIX) product., Methods: Regression analysis linked FIX activity to bleed rates. Pharmacokinetic parameters were used to estimate FIX activity over time. The probability of bleeds was estimated for both treatment arms. A Markov model estimated the presence of target joints and annualized bleed rates (ABRs)., Results: Higher FIX activity showed reduced ABRs (p < 0.001). Target joints resulted in higher bleed rates (p < 0.001). When FIX activity levels and bleed risks were applied to the Markov model, ABRs for nonacog beta pegol and its comparator were 2.40 and 6.36, respectively., Conclusion: This model provides a starting point for assessing the added value of new FIX products.
- Published
- 2018
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